Washington, DC – The March approval of ITF Therapeutics’ Duvyzat marked the third new FDA-approved treatment in 10 months for Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness and degeneration. In June 2023, the regulator greenlit Sarepta’s Elevidys as the first-ever gene therapy for DMD. That...
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Tosse, France – After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals. Glioblastoma, a deadly brain cancer, is notoriously difficult to...
Toronto, Canada – After a demoralizing period punctuated by the withdrawal of one of the few marketed therapies for ALS, investment in new biotechs, state-backed collaborative initiatives and buzz at BIO2025 suggest a new day in drug development for one of medicine’s most intractable diseases. The past couple of years...
Basel, Switzerland – Novartis on Saturday posted two promising Phase III readouts in rare kidney diseases, setting the pharma up for two potential approvals in the space. The company presented these data at the European Renal Association Congress over the weekend. Results from the Phase III ALIGN study showed that...
Des Moines, Iowa – Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline. Late last week, the world heard an incredible story: a baby with an exceptionally rare genetic...
Santa Cruz, Calif. – A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications. They’re rare, they’re confounding, and R&D for therapies to address them is painfully underfunded. Rare...
Washinton, DC – On March 15, the FDA’s Oncologic Drugs Advisory Committee will meet to discuss two CAR T cell therapies for multiple myeloma—Abecma, from Bristol Myers Squibb and 2seventy bio, and Carvykti, from Johnson & Johnson and Legend Biotech. Both treatments are currently approved for the treatment of adults...
Washington, DC – In February 2024, Larimar Therapeutics released positive Phase II data for its injectable subcutaneous investigational agent nomlabofusp in treating Friedreich’s ataxia, a rare disease that causes neuromuscular degeneration. The data indicate that Larimar could go head-to-head in the market with Biogen’s Skyclarys, the only disease-specific therapy for Friedreich’s...
Washington, DC – As the oncology space moves away from single oncogene-targeted treatments, immunotherapies that aim to prevent immune fatigue and enhance immune cell engagement are emerging as promising therapeutic trends, experts told BioSpace. But these therapies are not always intended to be deployed on their own. Increasingly, scientists and...
Dallas, Texas – Taysha Gene Therapies touted positive early-stage data last month for its gene therapy to treat Rett syndrome, which has become the company’s main focus after cutting other programs and laying off 35% of its staff. If successful, the candidate for Rett syndrome has the capability to put...